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IOL News
30-05-2025
- Health
- IOL News
Alyssa's fight: Parents launch campaign for urgent treatment of rare condition
Somerset Park couple raises funds for daughter diagnosed with MPS I. Image: Supplied A young couple from Somerset Park, KwaZulu-Natal, has launched a desperate effort to secure life-saving treatment for their infant daughter, Alyssa Cas Aldworth, after she was diagnosed with a rare and potentially fatal genetic disorder. Alyssa, born on September 28, 2024, was recently diagnosed with Mucopolysaccharidosis Type 1 (MPS I), a rare condition caused by the absence of the IDUA gene. The gene is responsible for producing an enzyme that breaks down complex sugars in the body. Without treatment, children with MPS I rarely live past the age of ten. Her father, 29-year-old Trysten Aldworth, described the news as devastating. "Hearing that your only daughter has a rare genetic disorder with an extremely high mortality rate has broken us beyond words," he said. "It's something no parent should ever have to hear." Alyssa, affectionately known as Ally, spent her first 10 days in a neonatal intensive care unit due to severe heart and lung complications. Despite initial health setbacks, she has continued to show signs of improvement with regular medical monitoring. The condition requires immediate medical intervention in the form of Enzyme Replacement Therapy (ERT), using a drug called laronidase (Aldurazyme). However, the therapy comes at a steep cost, quoted at R78,064.64 per month, excluding VAT and must be administered weekly for life. Even with ERT, the enzyme cannot cross the blood-brain barrier, limiting its effectiveness in halting neurological damage caused by MPS I. As a result, doctors are urgently considering a Hematopoietic Stem Cell Transplantation (HSCT), a more aggressive treatment aimed at halting disease progression if performed within the first two years of life. The cost of HSCT abroad is estimated between £150,000 and £200,000 (approximately R3.6 million to R4.9 million), though the family hopes to explore a more affordable option within South Africa. A consultation with a haematologist is scheduled this week to assess the feasibility of the treatment locally. Video Player is loading. Play Video Play Unmute Current Time 0:00 / Duration -:- Loaded : 0% Stream Type LIVE Seek to live, currently behind live LIVE Remaining Time - 0:00 This is a modal window. Beginning of dialog window. Escape will cancel and close the window. Text Color White Black Red Green Blue Yellow Magenta Cyan Transparency Opaque Semi-Transparent Background Color Black White Red Green Blue Yellow Magenta Cyan Transparency Opaque Semi-Transparent Transparent Window Color Black White Red Green Blue Yellow Magenta Cyan Transparency Transparent Semi-Transparent Opaque Font Size 50% 75% 100% 125% 150% 175% 200% 300% 400% Text Edge Style None Raised Depressed Uniform Dropshadow Font Family Proportional Sans-Serif Monospace Sans-Serif Proportional Serif Monospace Serif Casual Script Small Caps Reset restore all settings to the default values Done Close Modal Dialog End of dialog window. Advertisement Next Stay Close ✕ Ad loading The couple launched a campaign on Back a Buddy with the aim to raise R468,000 and have raised close to R206,000. Aldworth who has been married to his wife Jade for five years, said their community's support has been overwhelming. "In just days, the amount of people who have called, messaged, donated or shared Ally's story has been nothing short of amazing. It's scary, but in the best possible way." Ally's middle name, Cas, honours Jade's late father, Craig Andrew Suttie, who died in July 2023 from cerebral malaria at the age of 47. The family, together since 2013, remains hopeful as they navigate complex medical decisions. "We just want what every parent wants; for our child to be healthy, to live a full life, and one day walk down the aisle," Trysten added. "We're going to do everything in our power to make that possible." IOL News Get your news on the go, click here to join the IOL News WhatsApp channel.
Associated Press
13-05-2025
- Health
- Associated Press
Immusoft to Present Data at ASGCT 2025, Demonstrating Positive Results from the World's First Engineered B Cell in a Human Clinical Trial
Safety and initial activity of autologous human B cells engineered to express the enzyme iduronidase. Results from a first-in-human clinical trial in subjects with MPS I SAN FRANCISCO, May 13, 2025 /PRNewswire/ -- Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with diseases modifiable with therapeutic proteins, today announced that Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, will present recent clinical data at the American Society of Cell and Gene Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17, 2025, in New Orleans, LA. Immusoft will present the following: Oral Abstract Session: Gene Therapy Clinical Trials Title: Autologous human peripheral blood B cells genetically engineered to express human iduronidase: Results from a first-in-human clinical trial in subjects with mucopolysaccharidosis type I (MPS I) Date and Location: Wednesday, May 14th, 1:30 PM—3:15 PM CT, New Orleans Theater B. Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company's lead drug candidate, ISP-001, is a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease. ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA) for treating MPS I. In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene-modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy), both of which can be associated with severe toxicities. There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I, where the current standard of care is enzyme replacement therapy (ERT) or hematopoietic stem cell therapy transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous therapeutic indications. Additional information regarding the ASGCT presentation can be found at ASGCT. About Immusoft Immusoft is a cell therapy company focused on developing novel therapies for rare diseases using a sustained delivery of protein therapeutics from a patient's cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient's B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. The company is based in Seattle, WA. For more information, visit Contact Media Susan Roberts Roberts Communications 202-779-0929 [email protected] View original content to download multimedia: SOURCE Immusoft
